Abstract
Prion diseases are infectious and fatal neurodegenerative disorders of man and animals which are characterized by spongiform degeneration in the central nervous system. In human diseases, the manifestation can be sporadic, familial or acquired by infection. Prion disorders are caused by the accumulation of an aberrantly folded isoform of the cellular prion protein (PrPc), commonly named PrPSc. Although prion diseases are usually rare, they have the potential to be transferred within and also between species by infection processes, giving then raise even to epidemic scenarios. As pathology is obviously restricted to the central nervous system pre-mortem diagnosis is usually hard to achieve. Promising approaches towards the development of therapeutic and even prophylactic anti-prion regimens were recently made. However, only a profound knowledge of the infectious agent and its replication strategy enables the design of effective anti-prion strategies. Cell culture models were highly instrumental in uncovering fundamental aspects of prion propagation. In this chapter, the cellular and molecular biology of prion proteins in general is discussed and prophylactic and therapeutic concepts derived thereof are introduced. In particular, emphasis is put on strategies targeting PrPc which is absolutely needed as substrate for prion conversion, and on intrinsic cellular clearance mechanisms for prions.
Keywords: Prion diseases, prion protein, prion clearance, prion therapy, anti-prion drugs
Infectious Disorders - Drug Targets
Title: Therapy in Prion Diseases: From Molecular and Cellular Biology to Therapeutic Targets
Volume: 9 Issue: 1
Author(s): Carmen Krammer, Ina Vorberg, Hermann M. Schatzl and Sabine Gilch
Affiliation:
Keywords: Prion diseases, prion protein, prion clearance, prion therapy, anti-prion drugs
Abstract: Prion diseases are infectious and fatal neurodegenerative disorders of man and animals which are characterized by spongiform degeneration in the central nervous system. In human diseases, the manifestation can be sporadic, familial or acquired by infection. Prion disorders are caused by the accumulation of an aberrantly folded isoform of the cellular prion protein (PrPc), commonly named PrPSc. Although prion diseases are usually rare, they have the potential to be transferred within and also between species by infection processes, giving then raise even to epidemic scenarios. As pathology is obviously restricted to the central nervous system pre-mortem diagnosis is usually hard to achieve. Promising approaches towards the development of therapeutic and even prophylactic anti-prion regimens were recently made. However, only a profound knowledge of the infectious agent and its replication strategy enables the design of effective anti-prion strategies. Cell culture models were highly instrumental in uncovering fundamental aspects of prion propagation. In this chapter, the cellular and molecular biology of prion proteins in general is discussed and prophylactic and therapeutic concepts derived thereof are introduced. In particular, emphasis is put on strategies targeting PrPc which is absolutely needed as substrate for prion conversion, and on intrinsic cellular clearance mechanisms for prions.
Export Options
About this article
Cite this article as:
Krammer Carmen, Vorberg Ina, Schatzl M. Hermann and Gilch Sabine, Therapy in Prion Diseases: From Molecular and Cellular Biology to Therapeutic Targets, Infectious Disorders - Drug Targets 2009; 9 (1) . https://dx.doi.org/10.2174/1871526510909010003
DOI https://dx.doi.org/10.2174/1871526510909010003 |
Print ISSN 1871-5265 |
Publisher Name Bentham Science Publisher |
Online ISSN 2212-3989 |
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
- Announcements
Related Articles
-
Structure-Based Design, Synthesis and Molecular Modeling Studies of Thiazolyl Urea Derivatives as Novel Anti-Parkinsonian Agents
Medicinal Chemistry Editorial: In Silico Studies in Drug Research Against Neurodegenerative Diseases
Current Neuropharmacology Current Progresses on Nanodelivery Systems for the Treatment of Neuropsychiatric Diseases: Alzheimer’s and Schizophrenia
Current Pharmaceutical Design Invokana (Canagliflozin) as a Dual Inhibitor of Acetylcholinesterase and Sodium Glucose Co-Transporter 2: Advancement in Alzheimer’s Disease- Diabetes Type 2 Linkage via an Enzoinformatics Study
CNS & Neurological Disorders - Drug Targets Synergistic Two-Way Interactions of Dietary Polyphenols and Dietary Components on the Gut Microbial Composition: Is There a Positive, Negative, or Neutralizing Effect in the Prevention and Management of Metabolic Diseases?
Current Protein & Peptide Science Therapeutic Potential of Vasoactive Intestinal Peptide and its Receptors in Neurological Disorders
CNS & Neurological Disorders - Drug Targets Development and Applications of Non-HIV-Based Lentiviral Vectors in Neurological Disorders
Current Gene Therapy An Insight Into Mitochondrial Dysfunction and its Implications in Neurological Diseases
Current Drug Targets Nanoparticlized System: Promising Approach for the Management of Alzheimer’s Disease through Intranasal Delivery
Current Pharmaceutical Design Mn (III) Tetrakis (4-Benzoic Acid) Porphyrin Protects Against Neuronal and Glial Oxidative Stress and Death After Spinal Cord Injury
CNS & Neurological Disorders - Drug Targets Alcohol Drinking, Apolipoprotein Polymorphisms and the Risk of Cardiovascular Diseases
Current Neurovascular Research Nanotheranostics in Evidence Based Personalized Medicine
Current Drug Targets Recent Advances in Bioreactors in Tissue Engineering and Regenerative Medicine
Current Tissue Engineering (Discontinued) Brain-Derived Neurotrophic Factor (BDNF) has Proliferative Effects on Neural Stem Cells through the Truncated TRK-B Receptor, MAP Kinase, AKT, and STAT-3 Signaling Pathways
Current Neurovascular Research Inhibitory Mechanism of An Anticancer Drug, Bexarotene Against Amyloid β Peptide Aggregation: Repurposing Via Neuroinformatics Approach
Current Pharmaceutical Design Preclinical Studies and Clinical Trials with Mesenchymal Stem Cell for Demyelinating Diseases: A Systematic Review
Current Stem Cell Research & Therapy ADP-Ribosylated Proteins as Old and New Drug Targets for Anticancer Therapy: The Example of ARF6
Current Pharmaceutical Design Dissecting the Mechanisms of Thrombogenesis and Atherosclerosis for Neurodegenerative Disorders
Current Neurovascular Research Stem Cell-Derived Motor Neurons: Applications and Challenges in Amyotrophic Lateral Sclerosis
Current Stem Cell Research & Therapy Brain Innate Immunity in the Regulation of Neuroinflammation: Therapeutic Strategies by Modulating CD200-CD200R Interaction Involve the Cannabinoid System
Current Pharmaceutical Design